Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.

 

Posted on: 13 May 2013, source: EMA
The European Medicines Agency has recently published a draft guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy. Recent advances in basic and clinical research have opened new perspectives for future therapeutic options in Duchenne and Becker muscular dystrophy (DBMD). As most of the cases of Duchenne muscular dystrophy (DMD) have an onset in early childhood, while the onset of Becker muscular dystrophy (BMD) covers a broader age spectrum, specific difficulties have been identified that pertain to diagnostic criteria, age- and stage related clinical relevance and different safety aspects.

Posted on: 12 May 2013, source: Gene Therapy Office
The aim of the governmental Gene Therapy Office is to streamline the licensing and permit granting procedures for clinical gene therapy studies in The Netherlands and to provide more insight into the procedures for investigators. The Gene Therapy Office is primarily a service for professionals conducting clinical gene therapy research involving human subjects. The official website is www.loketgentherapie.nl.

Posted on: 28 March 2013, source: MedPageToday
Researchers reported encouraging but preliminary results of gene-based immune therapy in two children with relapsed and refractory acute lymphoblastic leukemia (ALL). Both children achieved a remission after they were given T cells modified to attack B cells bearing the surface protein CD19, according to Stephan A. Grupp, MD, PhD, of the Children's Hospital of Philadelphia, and colleagues.

Posted on: 28 February 2013, source: Lentigen
Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery products, announced today that that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to P140K methylguanine methyltransferase (MGMT) transduced human CD34 cells (product name: LG631-CD34) for bone marrow protection in the treatment of glioblastoma multiforme. Orphan drug designation qualifies Lentigen for seven years of market exclusivity following marketing approval by the FDA and provides other development-related incentives.

Posted on: 5 February 2013, source: Molecular Therapy
In the latest issue of the journal Molecular Therapy, Professor Jacques P. Tremblay (president of the Association of Gene Therapy of Quebec and researcher in the Research center of the Centre Hospitalier Universitaire (CHU) of Quebec) launches a call - with 50 other world experts in gene therapy - for the creation of an International Gene Therapy Consortium for Monogenic Diseases. The bases of this consortium will be established during a workshop, which will be held during the congress of the American Society of Gene and Cell Therapy (ASGCT) in Salt Lake City in May 2013.

Posted on: 1 February 2013, source: PlosBlogs
By Ricki Lewis - I am astonished, once again, by the complexity and unpredictability of science. Last week, a paper in the Proceedings of the National Academy of Science (PNAS) reported that gene therapy to treat a form of blindness called Leber congenital amaurosis type 2 (LCA2) doesn’t stop degeneration of the rods and cones – the photoreceptor cells that provide vision. Gene therapy sends genetic instructions for a protein called RPE65 into a layer of cells that supports the rods and cones – the retinal pigment epithelium, or RPE. The protein is essential for the eye to use vitamin A. And the gene therapy works, so far.